Top 8 Breakthrough Gene Therapies for Retinitis...

FDA Greenlights XOLREMDI Capsules from X4 Pharmaceuticals for WHIM Syndrome Treatment X4 Pharmaceuticals has declared that the FDA has granted approval for XOLREMDI™ (mavorixafor) capsules to be utilized in individuals aged 12 and above who have WHIM syndrome (characterized by warts, hypogammaglobulinemia, infec...

Ipsen Partners with Skyhawk Therapeutics for RNA-Focused Research in Rare Neurological Disorders Ipsen and Skyhawk Therapeutics have entered into an exclusive global partnership to explore and create new small molecules that can influence RNA for rare neurological conditions. Under this agreement, Ipsen has the ...

Roche's Columvi Achieves Primary Endpoint of Prolonged Overall Survival in Patients with Relapsed or Refractory Diffuse Large B-Cell Lymphoma in Phase III STARGLO Trial Roche reported that the Phase III STARGLO trial successfully achieved its main goal of improving overall survival. The research revealed that in...

As we step into the crisp corridors of 2024, the healthcare landscape unfolds a compelling saga of mergers, strategic funding, and transformative acquisitions. In this month-by-month analysis, we delve into the intricate tapestry of industry dynamics, exploring the impactful maneuvers that are shaping the healthcar...

The progress of mRNA-based therapeutics involves several key stages, including mRNA design, synthesis, entrapment, pharmacodynamics, pharmacokinetics, in vivo and in vitro safety evaluation, manufacturing, and clinical trials. Among these stages, mRNA design and synthesis play pivotal roles in developing mRNA-based...

A month following Novartis’ foray into the paroxysmal nocturnal hemoglobinuria (PNH) treatment domain, AstraZeneca strengthens its position in this field as its latest contender, Voydeya, secures a groundbreaking approval in Japan. Voydeya (danicopan), an innovative oral factor D inhibitor, has received approval fr...